| Primary | Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) | Open Label Treatment Period (OLTP) | Safety analysis set (SAF) included all randomized participants who received any amount of study drug and was based on the treatment received (as treated); Here 'number analyzed' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Through Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
| | | Title | Denominators | Categories |
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| Participants with any TEAE | | | | Participants with serious TEAE | | |
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| Secondary | Percent Change of Lactate Dehydrogenase (LDH) From Pre-treatment to End-of-treatment Period | OLTP Pre-treatment (mean of LDH values prior to combination dosing); End-of-treatment (mean of LDH value at week 24- through week 28); percentage of change in Upper Limit of Normal (xULN). | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Percentage of change | | End of treatment period, approximately 28 Weeks | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants Maintaining Adequate Control of Hemolysis From Baseline (Day 1) Through Week 28 | OLTP Adequate control of hemolysis is defined as LDH values ≤1.5 × Upper limit of normal (ULN) from baseline (day 1) to week 28 | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' is the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1) through Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants Maintaining Adequate Control of Hemolysis From Week 4 Through Week 28 | | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of Participants | | Week 4 through Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Adequate Control of Hemolysis at Each Visit Day 1 Through Week 28 | OLTP; Adequate control at a visit is defined as having LDH <=1.5 x ULN at that visit | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Day 1 through Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1) Through Week 28 | OLTP; Normalization of LDH was defined as LDH ≤1.0 × ULN at each visit | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'number analyzed' = number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1) through Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Average LDH (U/L) Based on Area Under the Curve (AUC) From OLTP Baseline (Day 1) Through Week 28 | | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Units per Liter (U/L) | | Baseline (Day 1) through Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Average LDH (U/L) Based on Area Under the Curve (AUC) From OLTP Week 4 Through Week 28 | | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | U/L | | Week 4 through Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Breakthrough Hemolysis From Baseline (Day 1) Through Week 28 | OLTP Breakthrough hemolysis is defined as an increase in LDH with concomitant signs or symptoms associated with hemolysis: • An increase in LDH occurs when:
- LDH ≥2 × ULN if pre-treatment LDH is ≤1.5 × ULN or
- LDH ≥2 × ULN after initial achievement of LDH ≤1.5 × ULN if pre-treatment LDH is >1.5 × ULN Signs or symptoms should correspond to those known to be associated with intravascular hemolysis due to Paroxysmal nocturnal hemoglobinuria (PNH) limited to the following: new onset or worsening fatigue, headache, dyspnea, hemoglobinuria, abdominal pain, scleral icterus, erectile dysfunction, chest pain, confusion, dysphagia, anemia including hemoglobin value significantly lower (ie, ≥2g/dL decrease) compared to patient's known baseline hemoglobin values, and thrombotic event.
| Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1) through Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Hemoglobin Stabilization From Baseline (Day 1) Through Week 28 | OLTP Hemoglobin stabilization was defined as participants who did not receive an RBC transfusion and had no decrease in hemoglobin level of ≥2 grams per deciLiter (g/dL). | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1) through Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in Hemoglobin Levels From Baseline (Day 1) Through Week 28 | | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | grams per Liter (g/L) | | Baseline (Day 1) to Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Transfusion Avoidance From Baseline (Day 1) Through Week 28 | OLTP Not requiring a red blood cell (RBC) transfusion as per protocol algorithm | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1) to Week 28 | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Rate of Red Blood Cells (RBCs) Transfused From Baseline (Day 1) to Week 28 | OLTP Rate of RBCs transfused is defined as number of events per person-years of treatment. For each participant, the participant-years are the time from first dose date to week 28 (or early terminations visit if subject discontinued the study early) in the OLTP. | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | 95% Confidence Interval | Events per person-years of treatment | | Baseline (Day 1) to Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Number of Per-Protocol RBC Units Transfused From Baseline (Day 1) Through Week 28 | | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Units | | Baseline (Day 1) to Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in Total Complement Hemolysis Activity Assay (CH50) From Baseline (Day 1) Through Week 28 | | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Units per milliliter (U/mL) | | Baseline (Day 1) to Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline (Day 1) Through Week 28 | OLTP FACIT fatigue is a 13-item scale and for each item 4 is not at all fatigued to 0 very much fatigued. Higher FACIT-Fatigue scores indicate less fatigue (scores range from 0-52). A 5-point change is considered clinically meaningful. | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline (Day 1) to Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in Global Health Status/Quality of Life Scale (GHS/QoL) on the European Organization for Research and Treatment of Cancer: Quality-of-Life Questionnaire Core 30 Items (EORTC QLQ-C30) From Baseline (Day 1) Through Week 28 | OLTP EORTC QLQ-C30 is a 30-question tool used to assess the overall quality of life (QoL) in cancer participants. Items contributing to the GHS/QoL, were scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that transformed score lies between 0 to 100. A higher score indicates better global health status/functioning and a negative change from baseline indicated less improvement. | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline (Day 1) to Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran |
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| Secondary | Change in Physical Function (PF) Scores on the EORTC QLQ-C30 From Baseline (Day 1) Through Week 28 | OLTP; EORTC QLQ-C30 is a 30-question tool used to assess the overall quality of life (QoL) in cancer participants. Items contributing to the GHS/QoL, were scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that transformed score lies between 0 to 100. A higher score indicates better global health status/functioning and a negative change from baseline indicated less improvement. | Full analysis set (FAS) = included all randomized participants who received any amount of study drug & had at least 1 post-baseline assessment; based on treatment allocated (as randomized); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline (Day 1) to Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Concentrations of Total Pozelimab in Serum on Week 28 | | The pharmacokinetic (PK) analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received. | Posted | | Mean | Standard Deviation | mg/L | | On Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Concentrations of Cemdisiran in Plasma on Week 28 | | The pharmacokinetic (PK) analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received. | Posted | | Mean | Standard Deviation | mg/L | | On Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Concentrations of Total C5 on Week 28 | | The pharmacokinetic (PK) analysis set includes all treated participants who received any amount of study drug (SAF) and who had at least 1 non-missing analyte measurement following the first dose of combination treatment. The PK analysis set is based on the actual treatment received. | Posted | | Median | Inter-Quartile Range | mg/L | | On Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Number of Participants With Pozelimab Anti-Drug Antibody (ADA) Responses Over Time | | Anti-Drug Antibodies (ADA) Analysis Set; Here 'n' = the number of evaluable participants at a certain timepoint | Posted | | Number | | Participants | | Up to Week 52 (OLTP [ Week 0 - Week 28] + OLEP [Week 28 - Week 52]) | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Number of Participants With Cemdisiran Anti-Drug Antibody (ADA) Responses Over Time | | Anti-Drug Antibodies (ADA) Analysis Set; Here 'n' = the number of evaluable participants at a certain timepoint | Posted | | Number | | Participants | | Up to Week 52 (OLTP [ Week 0 - Week 28] + OLEP [Week 28 - Week 52]) | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With TEAEs for Participants Who Received Treatment Intensification | OLTP No participants received dose intensification during the study; Therefore, assessment of the safety of pozelimab + cemdisiran combination therapy in participants requiring dose intensification was not conducted. | Safety analysis set (SAF) included all randomized participants who received any amount of study drug and was based on the treatment received (as treated); Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | | | | | Through Week 28 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change of LDH From Baseline (Day 1e) to Week 24e | Optional Open-Label Extension Period (OLEP) | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | U/L | | Baseline (Day 1e) to Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percent Change of LDH From OLEP Baseline (Day 1e) to Week 24e | OLEP; Percentage of change for units per liter (U/L) | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Percentage of change | | Baseline (Day 1e) to Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change of LDH From Baseline (Day 1e) to Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | U/L | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percent Change of LDH From Baseline (Day 1e) to Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Percentage of change | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants Maintaining Adequate Control of Hemolysis From Baseline (Day 1e) Through Week 24e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of Participants | | Baseline (Day 1e) through Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants Maintaining Adequate Control of Hemolysis From Baseline (Day 1e) Through Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of Participants | | Baseline (Day 1e) through Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Adequate Control of Hemolysis at Each Visit From Baseline (Day 1e) Through Week 52e | OLEP Adequate control at a visit is defined as having LDH <=1.5 x ULN at that visit | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1e) through Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Normalization of LDH at Each Visit From Baseline (Day 1e) Through Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1e) through week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Average LDH (U/L) Based on Area Under the Curve (AUC) From OLEP Baseline (Day 1e) Through Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | U/L | | Baseline (Day 1e) through Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Breakthrough Hemolysis From Baseline (Day 1e) Through Week 24e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of Participants | | Baseline (Day 1e) through Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Breakthrough Hemolysis From Baseline (Day 1e) Through Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1e) through Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Hemoglobin Stabilization From Baseline (Day 1e) Through Week 24e | OLEP Participants who did not receive RBC transfusion and had no decrease in hemoglobin levels | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1e) through Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Hemoglobin Stabilization From Baseline (Day 1e) Through Week 52e | OLEP Participants who did not receive RBC transfusion and had no decrease in hemoglobin levels | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Baseline (Day 1e) through Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in Hemoglobin Levels From Baseline (Day 1e) to Week 24e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | g/L | | Baseline (Day 1e) to Week 24e | | | | ID | Title | Description |
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| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in Hemoglobin Levels From Baseline (Day 1e) to Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | g/L | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Per-Protocol Transfusion Avoidance From Baseline (Day 1e) Through Week 24e | OLEP Not requiring a RBC transfusion as per protocol algorithm | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of Participants | | Baseline (Day 1e) through Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With Per-Protocol Transfusion Avoidance From Baseline (Day 1e) Through Week 52e | OLEP Not requiring a RBC transfusion as per protocol algorithm | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of Participants | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Rate of RBCs Transfused From Baseline (Day 1e) to Week 24e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | 95% Confidence Interval | Events per person-years of treatment | | Baseline (Day 1e) to Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Rate of RBCs Transfused From Baseline (Day 1e) to Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Number | 95% Confidence Interval | Events per person-years of treatment | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Number of Units of RBCs Transfused From Baseline (Day 1e) to Week 24e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Units | | Baseline (Day 1e) to Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Number of Units of RBCs Transfused From Baseline (Day 1e) to Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Units | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in CH50 From Baseline (Day 1e) to Week 16e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | U/mL | | Baseline (Day 1e) to Week 16e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
| |
| Secondary | Change in CH50 From Baseline (Day 1e) to Week 24e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint; No CH50 samples were collected at Week 24e | Posted | | | | | | Baseline (Day 1e) to Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
| |
| Secondary | Change in CH50 From Baseline (Day 1e) to Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | U/mL | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
| |
| Secondary | Percent Change in CH50 From Baseline (Day 1e) to Week 16e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint; Since all participants had 0 values at baseline for CH50, the percentage change was not appropriate and undefined. Therefore, this endpoint was not able to be calculated. | Posted | | Number | | Percentage of change | | Baseline (Day 1e) to Week 16e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
| |
| Secondary | Percent Change in CH50 From Baseline (Day 1e) to Week 24e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint; Since all participants had 0 values at baseline for CH50, the percentage change was not appropriate and undefined. Therefore, this endpoint was not able to be calculated. | Posted | | Number | | Percentage of change | | Baseline (Day 1e) to Week 24e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
| |
| Secondary | Percent Change in CH50 From Baseline (Day 1e) to Week 52e | | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint; Since all participants had 0 values at baseline for CH50, the percentage change was not appropriate and undefined. Therefore, this endpoint was not able to be calculated. | Posted | | Number | | Percentage of change | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
|---|
| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
| |
| Secondary | Change in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline (Day 1e) to Week 52e | OLEP; The FACIT-Fatigue is a 13-item, self-administered assessment of an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related QoL in participants with cancer and other chronic illnesses. The FACIT-Fatigue items are measured with a 5-point Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating less fatigue. A 5-point change is considered clinically meaningful. | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
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| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in GHS/QoL on the EORTC QLQ-C30 From Baseline (Day 1e) to Week 52e | OLEP; EORTC QLQ-C30 is a 30-question tool used to assess the overall quality of life (QoL) in cancer participants. Items contributing to the GHS/QoL, were scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that transformed score lies between 0 to 100. A higher score indicates better global health status/functioning and a negative change from baseline indicated less improvement. | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
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| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Change in PF Scores on the EORTC QLQ-C30 From Baseline (Day 1e) to Week 52e | OLEP; EORTC QLQ-C30 is a 30-question tool used to assess the overall quality of life (QoL) in cancer participants. Items contributing to the GHS/QoL, were scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that transformed score lies between 0 to 100. A higher score indicates better global health status/functioning and a negative change from baseline indicated less improvement. | OLEP FAS included all participants who participated in the OLEP who received any amount of study drug in the OLEP and had at least 1 post-baseline efficacy assessment in the OLEP; Here 'n' = the number of evaluable participants at a specified timepoint | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline (Day 1e) to Week 52e | | | | ID | Title | Description |
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| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Percentage of Participants With TEAEs Up to Week 52 | | The OLEP SAF includes all participants who participated in the OLEP who received any amount of study drug in the OLEP; Here 'n' = number of evaluable participants at a specified timepoint | Posted | | Number | | Percentage of participants | | Up to Week 52 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Concentrations of Total Pozelimab in Serum on Week 52 | | The OLEP pharmacokinetic (PK) analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP. | Posted | | Mean | Standard Deviation | mg/L | | On Week 52 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Concentrations of Total C5 on Week 52 | | The OLEP pharmacokinetic (PK) analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP. | Posted | | Median | Inter-Quartile Range | mg/L | | On Week 52 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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| Secondary | Concentrations of Cemdisiran in Plasma on Week 52 | | The OLEP pharmacokinetic (PK) analysis set includes all participants who participated in the OLEP who received any amount of study drug in the OLEP and who had at least 1 non-missing analyte measurement following the first dose of study drug in the OLEP. | Posted | | Mean | Standard Deviation | mg/L | | On Week 52 | | | | ID | Title | Description |
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| OG000 | Pozelimab Q2W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 2 weeks (Q2W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment (pozelimab Q2W + cemdisiran) was administered on day 1 of the open-label treatment period (OLTP). In the open- label extension period (OLEP), participants received a regimen of pozelimab + cemdisiran, regardless of their treatment assignment in the OLTP. | | OG001 | Pozelimab Q4W + Cemdisiran | Pozelimab administered by subcutaneous (SC) injection every 4 weeks (Q4W) and cemdisiran administered by subcutanous (SC) injection. The first dose of the combination treatment was administered on day 1 of the OLTP. In the OLEP, participants received a regimen of pozelimab Q4W + cemdisiran, regardless of their treatment assignment in the OLTP. |
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